ChromaDex has announced that its treatment candidate nicotinamide riboside chloride (NRC) for the rare disease ataxia telangiectasia, has been granted US Food and Drug Administration (FDA) orphan drug and rare paediatric disease status.

The Los Angeles, California-based dietary supplement and food ingredient company has set plans in motion to file an FDA investigational new drug (IND) for NRC in this indication. As per the 7 June press release, the potential clinical studies will be led by ChromaDex scientific advisor Dr. Vilhelm Bohr.

Ataxia telangiectasia is a rare, inherited, progressive childhood disorder for which symptoms manifest neurologically and immunologically. The disease impacts a part of the brain that regulates motor function, with affected children subsequently exhibiting progressive difficulty with movement coordination and gait (ataxia), impaired eye coordination (oculomotor apraxia), and involuntary movement (choreoathetosis). There are no existing FDA-approved drugs for the treatment of this condition.

ChromaDex is not the only player in the ataxia telangiectasia treatment space. Earlier this week, San Francisco, California-based biotech Quince Therapeutics was awarded an FDA fast track designation for its  candidate EryDex. The drug-device treatment is being developed using autologous intracellular drug encapsulation (AIDE) technology and administers the glucocorticoid agonist dexamethasone sodium phosphate into a patient’s red blood cells.

EryDex is currently being evaluated in the Phase III NEAT study (NCT06193200). The trial was initially put on a partial hold, which was lifted in October 2023. The clinical hold was removed after the agency requested additional information on the plastics used in the single-use EryKit which is integral to the treatment. Quince also made other changes that ensured that the commercial version of EryKit complied with European regulations.

Back in 2022, Acasti Pharma also reported preliminary topline results from its Phase I pharmacokinetic study of its drug candidate GTX-102 for the treatment of ataxia telangiectasia. However, as per a June 2023 press announcement, the company announced the decision to evaluate strategic alternatives to maximise the value of GTX-102, which became a deprioritised pipeline asset.

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